Welcome to Gene Therapy Net
Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.
Updates on gene therapy for ‘bubble boy’ disease and cellular immunotherapy at ASGCT
Posted on: 15 May 2022, source: ASGCT
St. Jude Children’s Research Hospital scientists will discuss research on cellular therapies for pediatric cancer and more at the annual gathering of the professional society for gene and cellular therapy researchers.
St. Jude Children’s Research Hospital scientists will discuss research on cellular therapies for pediatric cancer and more at the annual gathering of the professional society for gene and cellular therapy researchers.
AAV Durability: The Road to a One-Shot Cure
Posted on: 15 April 2022, source: Hansonwade
Inconsistent long-term clinical data has highlighted the need to better understand the durability of AAV gene therapy. At Hanson Wade Intelligence, our Market Research division, we have classified 121 AAV gene therapy trials into 4 distinct groups based on the target tissue type – liver, muscle, CNS and ocular tissues. This allowed us to summarise available data on clinical durability as well as the key findings we observed across tissues, trials, doses and serotypes. We hope that this report will provide gene therapy developers with valuable insights on the durability of the current generation of AAV gene therapies.
Inconsistent long-term clinical data has highlighted the need to better understand the durability of AAV gene therapy. At Hanson Wade Intelligence, our Market Research division, we have classified 121 AAV gene therapy trials into 4 distinct groups based on the target tissue type – liver, muscle, CNS and ocular tissues. This allowed us to summarise available data on clinical durability as well as the key findings we observed across tissues, trials, doses and serotypes. We hope that this report will provide gene therapy developers with valuable insights on the durability of the current generation of AAV gene therapies.
Gene Therapy Sector Needs Better Cell Lines for Vector Production
Posted on: 4 April 2022, source: Genengnews
Gene therapy developers targeting diseases requiring high dose therapies face a challenge. Current vector production cell lines and genetic manipulation techniques are too costly for large-scale manufacturing, according to new research. The study, by scientists at Portugal’s Instituto de Biologia Experimental e Tecnológica (iBET), looked at areas of gene therapy production where innovation is needed.
Gene therapy developers targeting diseases requiring high dose therapies face a challenge. Current vector production cell lines and genetic manipulation techniques are too costly for large-scale manufacturing, according to new research. The study, by scientists at Portugal’s Instituto de Biologia Experimental e Tecnológica (iBET), looked at areas of gene therapy production where innovation is needed.
Gene Therapy Turns to the Use of Synthetic Biology Principles
Posted on: 4 March 2022, source: Genengnews
A new university spinout company is hoping to use synthetic biology to transform gene therapy manufacture. SynGenSys, from the University of Sheffield, plans to tap into engineering design principles to create synthetic promoters to target therapeutic genes to the correct patient tissues. “Typically, people use viral promoters, such as cytomegalovirus as their go-to promoter, but it’s a really complex piece of DNA active in many human tissues,” says David C. James, PhD, professor of bioprocess engineering and co-founder of SynGenSys.
A new university spinout company is hoping to use synthetic biology to transform gene therapy manufacture. SynGenSys, from the University of Sheffield, plans to tap into engineering design principles to create synthetic promoters to target therapeutic genes to the correct patient tissues. “Typically, people use viral promoters, such as cytomegalovirus as their go-to promoter, but it’s a really complex piece of DNA active in many human tissues,” says David C. James, PhD, professor of bioprocess engineering and co-founder of SynGenSys.
Lentiviral vector-based gene therapy demonstrates long-term safety and efficacy for Wiskott-Aldrich Syndrome
Posted on: 11 February 2022, source: MedicalXpress
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.
Nanoscope Therapeutics announces FDA clearance of IND for MCO-010 gene therapy in Stargardt patients
Posted on: 26 January 2022, source: Ophthalmology Times
Nanoscope Therapeutics Inc. announced that it has received IND clearance from the FDA to begin a Phase 2 trial of its Multi-Characteristic Opsin (MCO-010) ambient-light activatable optogenetic monotherapy to restore vision in Stargardt patients.
Nanoscope Therapeutics Inc. announced that it has received IND clearance from the FDA to begin a Phase 2 trial of its Multi-Characteristic Opsin (MCO-010) ambient-light activatable optogenetic monotherapy to restore vision in Stargardt patients.
Avrobio stops work on rare disease gene therapy after unexpected study results
Posted on: 6 January 2022, source: Biopharmadive
Avrobio, a Cambridge, Massachusetts-based gene therapy developer, will unexpectedly stop work on one of its most advanced treatments, citing disappointing results from a clinical trial that contrast with earlier, more promising findings. In a statement Tuesday, Avrobio said it would stop recruiting patients into a mid-stage study testing its gene therapy for Fabry disease, a rare inherited disorder, and deprioritize the research program. Monitoring will continue for the 14 Fabry patients who have received Avrobio's therapy so far.
Avrobio, a Cambridge, Massachusetts-based gene therapy developer, will unexpectedly stop work on one of its most advanced treatments, citing disappointing results from a clinical trial that contrast with earlier, more promising findings. In a statement Tuesday, Avrobio said it would stop recruiting patients into a mid-stage study testing its gene therapy for Fabry disease, a rare inherited disorder, and deprioritize the research program. Monitoring will continue for the 14 Fabry patients who have received Avrobio's therapy so far.
Gene Therapy News
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PTC's first gene therapy gains European regulator's backing - BioPharma Dive
May 20, 2022 | 18:12 pm
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Francis Collins Urges Gene Therapy Community to Scale Efforts to Tackle Rare Diseases - Genetic Engineering & Biotechnology News
May 18, 2022 | 19:20 pm
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With lead gene therapy nearing the clinic, SwanBio leans heavily on Syncona to close $56M series B - FierceBiotech
May 18, 2022 | 14:35 pm
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Aiming to be biggest biotech in Boston, Vertex plots 500-person cell, gene therapy R&D center - FierceBiotech
May 17, 2022 | 15:17 pm
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Study suggests postnatal gene therapy could treat Pitt-Hopkins syndrome - Drug Target Review
May 12, 2022 | 11:03 am
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